Rocket Pharma stocks cardiac gene therapies through $53 million acquisition of Renovacor

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Gene therapy developer Rocket Pharmaceuticals is on track to gain FDA approval for its pioneering program, but it’s also interested in preserving its pipeline inventory. The company agreed to Acquisition of Renovacora biotechnology working to develop a gene therapy for a form of hereditary heart failure.

According to financial terms announced Tuesday, the all-share transaction represents an equity value of $53 million, or an implied value of $2.60 per Renovacor share. That’s a 36.8% premium to Renovacor’s closing share price on Monday, but a sharp drop from last year, when the company went public via SPAC merger Her new shares saw her shares trade on the New York Stock Exchange at more than $10 a piece.

Renovacor in Cambridge, Massachusetts is developing gene therapies for cardiovascular disease caused by genetic factors. company appeared in 2019 With a $11 million funding round of a Series 1 preclinical development of a gene therapy that addresses BLCL2-associated atanogene 3 (BAG3) mutations that lead to dilated cardiomyopathy, a severe form of heart failure. Renovacor was founded by Arthur Feldman, a cardiologist and professor of medicine at Temple University.

Lead Renovacor REN-001 uses adeno-associated virus (AAV) to deliver a healthy copy of the BAG3 gene to cells. In preclinical testing, Renovacor said its gene therapy resulted in production of the functional BAG3 protein and improved heart function. Human testing is the next step. The company said it expects to submit a new research application for the drug in the second half of this year to support phase 1/2 clinical trials. Renovacor’s pipeline includes discovery-stage gene therapies that address BAG3 mutations as well as the expression and function of this gene. The company has also expanded its research to include cardiomyopathy caused by genetic arrhythmias.

“The acquisition of Renovacor aligns with our strategy to expand our leadership position in AAV-based gene therapy for heart disease and presents us with an ideal opportunity to continue our mission to transform the lives of heart failure patients through the power of gene therapy,” said Rocket CEO, Gaurav Shah, in a prepared statement.

Rocket’s cardiac gene therapy research focuses on Dannon’s disease, a heart muscle impairment caused by mutations in the LAMP2 gene. The Danone RP-A501 software of Cranberry, NJ, is currently in the first phase of testing. In a research note sent to investors, William Blair analyst Raju Prasad said the prevalence of BAG3-related dilated cardiomyopathy is estimated at 30,000 patients in the United States, a number that is expected to grow with more genetic testing and disease awareness. He added that Renovacor brings synergy to Rocket, as both companies use AAV-9 vectors to pursue genetically specific targets.

Rocket’s most advanced program, RP-L201, has reached a pivotal phase two of testing for leukopenia-1 (LAD-1), a rare disorder caused by mutations in the gene that encodes CD18, a protein that helps white blood cells attach to blood vessels. . Babies born with LAD-1 are susceptible to fungal and bacterial infections that can be life-threatening. in May, Rocket reported data showing 100% survival in seven patients 12 months after gene therapy infusion. In its report on financial results for the second quarter of 2022 last month, Rocket said it expects to file for Food and Drug Administration approval of its LAD-1 gene therapy in the first half of 2023.

The boards of both Rocket and Renovacor have approved the takeover, but shareholder approval for both companies is still needed. The deal is expected to close by the first quarter of next year.

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